BioMarin gets go-ahead to start gene therapy clinical trials
BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program.
BioMarin Pharmaceutical, a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, has announced that both the FDA and the Medicines and Healthcare Products Regulatory Agency (MHRA) have granted the company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307.
BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.
The company expects to start dosing patients in PHEARLESS, a Phase I/II study, in the first quarter of 2020 with product made at commercial scale from its award-winning gene therapy manufacturing facility. The company is actively preparing regulatory submissions to open additional clinical sites in other countries. BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program. Both the FDA and European Medicines Agency (EMA) have granted BMN 307 Orphan Status.
BMN 307 follows BioMarin's first investigational gene therapy clinical program: valoctocogene roxaparvovec to treat severe hemophilia A, currently in Phase III. Based on data from an interim analysis of the ongoing Phase III study, the EMA validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec, and the review process has begun. The company also has submitted a Biologics License Application to the FDA and anticipates the review to begin in February 2020.
"With BMN 307, we are joining together our expertise in PKU biology and the knowledge we have gained from developing the only two approved therapies for PKU with our understanding of gene therapy clinical development and manufacturing from our valoctocogene roxaparvovec experience," said Hank Fuchs, President, Worldwide Research and Development at BioMarin. "BioMarin has stood with the PKU community for over 15 years and remains dedicated to continuing to increase the body of medical knowledge in this devastating disease."
Related News
-
News Moderna to buy OriCiro Genomics in USD $85 million deal
Pharmaceutical company Moderna acquires genomics services company OriCiro Genomics, based in Tokyo, Japan, to boost mRNA therapeutics development and manufacturing portfolio. -
News New year, new prices: at least 350 drugs in USA to see price increases in January
Drugmakers such as Pfizer, GSK, BMS, AstraZeneca, and Sanofi are planning to raise prices for at least 350 unique drugs in the United States in early January. -
Sponsored Content 2023 Pharma Trend Outlook: Innovation, Resilience, and Pharma 4.0
Download our 2023 Pharma Trends Outlook report to discover the trends set to shape the pharmaceutical landscape in the new year, with expert opinions and insight from across the pharmaceutical value chain. -
News Strategic partnership between AstraZeneca and G42 Healthcare to increase pharmaceutical manufacturing in Abu Dhabi
A comprehensive, long-term partnership agreement between AstraZeneca and G42 Healthcare is set to enhance local drug manufacturing in Abu Dhabi, UAE, and bolster pharmaceutical manufacturing innovation within the country. -
News CordenPharma joins sustainability initiative to achieve Net Zero
The CDMO has joined the environmental initiative looking to align private sector companies with those aims upheld by the Paris Agreement to reduce global warming. -
News AstraZeneca acquires Neogene in shift into cell-based cancer therapies
AstraZeneca has recently announced that it will be acquiring the biotechnology company Neogene Therapeutics as part of it’s expansion into cell-based cancer treatments.
-
News A mRNA universal flu vaccine is in early stages of development
Researchers from the University of Pennsylvania have developed a universal flu vaccine using nanoparticle encapsulated mRNA. -
News Biovac partnership to increase African vaccine manufacturing capabilities
South African biopharma company Biovac signs a partnership deal with the International Vaccine Institute to develop and manufacture an oral cholera vaccine for both the African and global market.
Position your company at the heart of the global Pharma industry with a CPHI Online membership
-
Your products and solutions visible to thousands of visitors within the largest Pharma marketplace
-
Generate high-quality, engaged leads for your business, all year round
-
Promote your business as the industry’s thought-leader by hosting your reports, brochures and videos within your profile
-
Your company’s profile boosted at all participating CPHI events
-
An easy-to-use platform with a detailed dashboard showing your leads and performance